Navigation Links
Ultragenyx Announces Phase 1 Results of UX001 in Hereditary Inclusion Body Myopathy (HIBM), a Rare Neuromuscular Disease
Date:5/1/2012

ng from HIBM," said Emil D. Kakkis, MD, PhD, Chief Executive Officer of Ultragenyx. "Based on these favorable Phase 1 results, we look forward to moving quickly to initiate a Phase 2 study of UX001." 

Ultragenyx plans to present the Phase 1 data at the 17th Annual International World Muscle Society Congress in Perth, Australia later this year.

About HIBM  

HIBM is also known as GNE myopathy, distal myopathy with rimmed vacuoles (DMRV) and Nonaka disease.  HIBM is a severe, adult-onset, progressive, genetic neuromuscular disease caused by a deficiency of an enzyme in the first step of sialic acid biosynthesis needed for the modification of proteins and fats. Patients with HIBM typically begin to have weakness and abnormal walking at 18 to 30 years of age. Over the ensuing 10 to 20 years, many patients progressively lose significant functional ability and become wheelchair-bound. There are no current treatments for this disease.

About Ultragenyx

Ultragenyx is a privately held, developmental stage biotechnology company committed to bringing life-enhancing therapeutics for patients with rare and ultra-rare genetic diseases, also known as orphan and ultra-orphan diseases, to market.  The company focuses on rare metabolic diseases that affect small numbers of patients, but for which the unmet medical need is high and there are no effective treatments. Ultragenyx intends to build a sustainable pipeline of safe and effective therapies to address these underserved diseases.  Ultragenyx' lead program, UX001, is being evaluated as a potential treatment for hereditary inclusion body myopathy (HIBM), also known as GNE myopathy. 

The company is led by an experienced management team in rare disease therapeutics.  Ultragenyx is striving toward an improved model for successful rare disease drug development which has the potential to increase efficiency while maintaining appropriate sa
'/>"/>

SOURCE Ultragenyx Pharmaceutical Inc.
Copyright©2010 PR Newswire.
All rights reserved

Page: 1 2 3

Related medicine technology :

1. Ultragenyx Relocates Company Headquarters to Support Operational Expansion
2. Ultragenyx Granted Orphan Designations in Europe for Two Lead Product Candidates, UX001 for HIBM and UX003 for MPS 7
3. Ultragenyx Appoints Tom Kassberg as Chief Business Officer and Announces Management Team
4. Ultragenyx Granted Orphan Drug Designation for UX001 SA-ER for the Treatment of Hereditary Inclusion Body Myopathy (HIBM)
5. Ultragenyx Initiates Phase 1 Clinical Study of UX001 in Hereditary Inclusion Body Myopathy (HIBM), a Rare Muscle Wasting Disease
6. Acella Pharmaceuticals Announces a New Addition to the Commercial Team
7. Avanir Pharmaceuticals Announces Date Of Fiscal 2012 Second Quarter Financial Results And Conference Call
8. Endo Reports First Quarter Financial Results And Announces Return To Steady Supply Of OPANA® ER And Voltaren® Gel
9. Rigel Announces First Quarter 2012 Financial Results
10. National Jewish Health, the Nations Premier Respiratory Hospital, Announces Lung Cancer Screening Trial Using Blood Test and CT
11. Jazz Pharmaceuticals Announces First Quarter 2012 Financial Results Conference Call On May 8, 2012
Post Your Comments:
*Name:
*Comment:
*Email:
(Date:4/27/2015)... , April 27, 2015   AMC Health , ... showcase advances in monitored care that are transforming chronic ... ATA 2015 Conference, May 2-5 at the ... Health will share information about how their innovative ... help transform and personalize healthcare delivery at Booth #2116 ...
(Date:4/27/2015)... NEW YORK , April 27, 2015 ... claims against the board of directors of Aerie Pharmaceuticals, ... concerning whether the board has breached its fiduciary duties ... Company issued a press release announcing results from its ... things, the press release disclosed that in the trial, ...
(Date:4/27/2015)... 2015  USL di Reggio Emilia, the Regional Health ... has implemented Zebra Technologies Corporation (NASDAQ: ... Wireless Network Solution (WNS) provided by Zebra has ... in real time via mobile computers, contributing to improved ... also provides employee and guest Wi-Fi access and enables ...
Breaking Medicine Technology:AMC Health to Showcase Advances in Monitored Care at ATA 2015 2AMC Health to Showcase Advances in Monitored Care at ATA 2015 3AMC Health to Showcase Advances in Monitored Care at ATA 2015 4Harwood Feffer LLP Announces Investigation of Aerie Pharmaceuticals, Inc. 2Zebra Technologies' Wireless Solutions Help Reggio Emilia Hospital Staff Improve Patient Care and Reduce Costs 2Zebra Technologies' Wireless Solutions Help Reggio Emilia Hospital Staff Improve Patient Care and Reduce Costs 3Zebra Technologies' Wireless Solutions Help Reggio Emilia Hospital Staff Improve Patient Care and Reduce Costs 4Zebra Technologies' Wireless Solutions Help Reggio Emilia Hospital Staff Improve Patient Care and Reduce Costs 5
... NEW YORK, Nov. 9 /PRNewswire-Asia-FirstCall/ -- American Oriental,Bioengineering, Inc. ... pharmaceutical,company dedicated to improving health through the development, manufacture ... the counter ("OTC"),products, today announced that it plans to ... 2009, after the market closes. , ...
... 9, 2009 Arena Pharmaceuticals, Inc. (Nasdaq: ARNA ) today ... , Arena reported a lower net loss allocable to common stockholders ... per share, compared to a net loss allocable to common stockholders ... per share, and a net loss allocable to common stockholders in ...
Cached Medicine Technology:Arena Pharmaceuticals Announces Third Quarter 2009 Financial Results and Recent Developments 2Arena Pharmaceuticals Announces Third Quarter 2009 Financial Results and Recent Developments 3Arena Pharmaceuticals Announces Third Quarter 2009 Financial Results and Recent Developments 4Arena Pharmaceuticals Announces Third Quarter 2009 Financial Results and Recent Developments 5Arena Pharmaceuticals Announces Third Quarter 2009 Financial Results and Recent Developments 6Arena Pharmaceuticals Announces Third Quarter 2009 Financial Results and Recent Developments 7Arena Pharmaceuticals Announces Third Quarter 2009 Financial Results and Recent Developments 8Arena Pharmaceuticals Announces Third Quarter 2009 Financial Results and Recent Developments 9
(Date:4/27/2015)... April 27, 2015 According to a ... Craniofacial Research), after chronic back pain, TMJ disorders are ... in pain and disability. In addition, the report revealed ... of the population, costing an estimated 4 billion dollars ... Americans suffering from seemingly unrelated symptoms such as headaches, ...
(Date:4/27/2015)... April 27, 2015 The Doctors on ... connect clients of personal injury attorneys with the best doctors ... has seen steady growth throughout their history and that trend ... DC and his team in the Tujunga / ... doctor and massage therapist, patients can find comprehensive care and ...
(Date:4/27/2015)... Lambertville, NJ (PRWEB) April 27, 2015 ... consulting firm, is proud to announce their sponsorship ... and Warren Counties. Girls on the Run ... in third through eighth grade. The program is ... lessons with uplifting workouts aimed to develop the ...
(Date:4/27/2015)... Ticket Down is a reputable source ... Valley Music Theatre and the Isleta Amphitheater. ... Mexico area can celebrate because their favorite performer will ... Buffett will be performing alongside legendary performers, Huey Lewis ... during the summer and fall months of 2015 are ...
(Date:4/27/2015)... York, NY (PRWEB) April 27, 2015 ... are creating a seamless referral process for the Health ... with chronic heart failure or diabetes, for instance, may ... most primary physicians coordinate with many other care providers. ... referring hundreds of thousands of patients each year, the ...
Breaking Medicine News(10 mins):Health News:Dentist in Graham NC Reveals that a Jaw Disorder May Be the Cause of Chronic Headaches and Back Pain 2Health News:Prominent Doctors with Distinguished Backgrounds Join Doctors on Liens in Tujunga 2Health News:TayganPoint Consulting Group Announces Sponsorship of Girls on the Run 2Health News:Cheap Jimmy Buffett Tickets at Alpine Valley Music Theatre in Elkhorn, WI and at Isleta Amphitheater in Albuquerque, NM Now on Sale at TicketDown.com 2Health News:Mount Sinai Launches System for Faster Referrals 2Health News:Mount Sinai Launches System for Faster Referrals 3
... , , WINSTON-SALEM, N.C., July 17 ... for finance and chief financial officer of Wake Forest University Baptist ... Center reorganization that began two years ago. He began work July ... the Medical Center. , , ...
... remain volatile issues in the drive for bipartisan support , ... to overhaul the nation,s health-care system started to emerge from ... that this may, at long last, be the year that ... health care more affordable. , "I think we,re in a ...
... peer-reviewed scientific journal- , , BELLEVILLE, ON, July ... a research-based, technology-driven Canadian biopharmaceutical company, today announced that ... with the Company,s Escherichia coli (E. coli) O157 vaccine ... issue of Foodborne Pathogens and Disease (Vol. 6, Number ...
... ... network of healthcare associations to address high workforce shortage of lab professionals , ... Washington DC (PRWEB) July ... medical society based in Chicago, today announced it is joining the National Healthcare Career ...
... ... expert on searchbodydetox.com, a new informational Web site devoted to all aspects of whole body ... Potomac, ... alternative health movement, is now a featured expert on searchbodydetox.com , a new informational ...
... for protecting female partner, researchers say , THURSDAY, July ... HIV from infected men to women, says a study ... men, who were uncircumcised before the start of the ... circumcision after two years. The study also included HIV-uninfected ...
Cached Medicine News:Health News:Wake Forest Baptist Medical Center Names Chadwick CFO 2Health News:Congress Braces for Showdown Over Health-Care Reform 2Health News:Congress Braces for Showdown Over Health-Care Reform 3Health News:Econiche(TM) Vaccine Efficacy Summarized in July Issue of Foodborne Pathogens and Disease 2Health News:Econiche(TM) Vaccine Efficacy Summarized in July Issue of Foodborne Pathogens and Disease 3Health News:Econiche(TM) Vaccine Efficacy Summarized in July Issue of Foodborne Pathogens and Disease 4Health News:Econiche(TM) Vaccine Efficacy Summarized in July Issue of Foodborne Pathogens and Disease 5Health News:Econiche(TM) Vaccine Efficacy Summarized in July Issue of Foodborne Pathogens and Disease 6Health News:American Society for Clinical Pathology Joins the National Healthcare Career Network 2Health News:American Society for Clinical Pathology Joins the National Healthcare Career Network 3Health News:Dr. Julian Whitaker Featured on Searchbodydetox.com 2Health News:Circumcision Doesn't Lessen HIV Transmission 2
Angled for temporal approach. Open 14 mm rounded blades for patient comfort. Dull finish....
Open wire style blades allow complete manipulation of the phaco tip inside the eye. Lid control is excellent, exposure is maximized, and the likelihood of positive pressure is reduced....
Pediatric model. Blades: 10 mm. Solid blades with locking mechanism. Dull finish....
Fenestrated 16 mm blades. Self-locking mechanism. Dull finish....
Medicine Products: